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Portfolio management strategies for growth and impact in cells and genes

Vaseline 2 months ago

In recent years, cell and gene therapies have taken center stage in biotechnology as they promise to address a wide range of indications and patient populations that have historically had significant unmet needs. Even despite a decline in investment, the cell and gene therapy industry saw $11.7 billion in investments in 2023. Building on these current trends and wins, McKinsey has highlighted the cell and gene therapy industry as a key opportunity that will continue to attract strong opportunities. interest from investors.

With more than 2,500 existing developers worldwide, and many more looking to enter this sector, stakeholders and investors are looking for ways to navigate and succeed in this promising yet competitive sector. The promise of these treatments for both patients and investors continues to drive efforts in this area, but success in bringing cell and gene therapies to market depends on informed portfolio management decisions.

Asset assessment for portfolio management

It is becoming increasingly difficult to stand out in the crowded cell and gene therapy market, making it crucial to choose the right candidates for commercial purposes. According to the latest industry reports, biotech companies pursuing opportunities in the cell and gene sector, with an emphasis on R&D initiatives, and companies with a robust clinical pipeline are well positioned to attract investor interest.

With 1,804 clinical trials underway and counting, strategic portfolio assessments and management are critical to success. Understanding the market potential and patient demand for a product is the first step. By analyzing disease prevalence, the competitive landscape, and the position of corresponding candidates in the development process, stakeholders can effectively allocate resources and prioritize the most promising therapies. A diverse portfolio is essential as it not only limits risk but also maximizes the chances of success while addressing a wide range of indications. Evaluating existing target indications, asset modalities and technologies in the pipeline can help assess how diversified the portfolio is.

When determining which agents to pursue, strong clinical data currently serves as one of the most powerful indicators of success in a product portfolio. Further assessing the scalability and feasibility of each candidate’s production can help gain clearer insights needed for strategic decision-making. Consider the complexity of the manufacturing process, the supply chain requirements of each process and the required regulatory path to market.

Opportunities for growth and impact in cells and genes

As the number of cell and gene therapy products in the pipeline continues to increase, there is growing recognition of the need to address larger patient populations and take into account economic and social considerations. Today, low- and middle-income patients continue to face significant health disparities and unmet medical needs. To address this, the industry must broaden its view of unmet needs and prioritize diseases that have a significant societal impact, even if they are not initially financially attractive. Refocusing R&D and investment efforts on the diseases most common in these patient populations can contribute to more equitable care and enable companies to have a significant impact on society while still achieving success in the sector.

When conducting a portfolio analysis, the most important considerations are the scalability and feasibility of production, the complexity of production processes and the requirements of the supply chain. Assessing these factors early in the development process can save time and money by avoiding costly delays and allows strategic allocation of resources to other promising programs. Looking to the future, industry leaders must continue to invest in emerging technologies such as gene editing, allogeneic cell therapy and non-viral delivery methods. By continually refining the pipeline based on patient demand and the most advanced technologies, biotech companies can remain at the forefront of cell and gene therapy development.

In the wake of the financial downturn, the industry is seeing a correction with increased merger and acquisition activity, along with rapid changes due to a highly supportive regulatory environment and picking up investor interest. As a result, leveraging real-time data, risk-based decision making and flexible portfolio management practices will be key to success. Furthermore, embracing flexibility will enable companies to quickly adapt to changes in the market and take advantage of emerging opportunities.

A holistic approach to portfolio management

While analyzing the program portfolio at the product level is important, aligning R&D activities and business strategy is critical for long-term success. Focusing efforts on opportunities with high market potential and patient impact, while taking into account the company’s resources and capabilities, can ensure strategic alignment with the company’s larger goals. A clear understanding of existing infrastructure and internal manufacturing expertise helps identify strengths to drive decision-making. Where internal capabilities are lacking, collaboration with external stakeholders is critical. In the growing field of cell and gene therapy, expertise and specialized skills are widely dispersed, making sharing resources and knowledge essential to advance the industry. Forming diverse teams and partnerships that complement existing capabilities can facilitate collective problem solving to shorten timelines, increase efficiencies, and ultimately improve patient outcomes.

As a crucial part of asset management, the financial implications must be analyzed through an in-depth cost-benefit analysis, taking into account development costs, potential income streams and return on investment. Forecasting models that take into account different market scenarios, investment landscapes and regulatory outcomes can help predict the performance of cell and gene therapy products. With thorough financial analysis, assessment of unique competitive advantages and deep insight into the pipeline, companies can position themselves to quickly overcome challenges and maximize the chances of success in bringing life-saving therapies to market.

As cell and gene therapies continue to make headlines as a major growth opportunity, success in this area will require both innovative technology and strategic foresight. Flexible portfolio management and an adaptable organization allow companies to take advantage of exciting opportunities while promoting resilience and growth, even in the face of market changes. Although highly competitive, the cell and gene therapy industry offers the opportunity for financial success and the chance to make an incredible difference in patients’ lives. With the right resources and strategy, companies can define success by driving innovation, addressing important unmet medical needs and ultimately improving patient outcomes.

Photo: Topp_Yimgrimm, Getty Images


Anshul Mangal is a biotech entrepreneur, experienced executive, board member, philanthropist and lawyer. He is the President of Project Farma (“PF”) and Precision for Medicine’s Manufacturing Solutions. He is also a board member of the Alliance for Regenerative Medicines, Alliance for mRNA Medicines and IQHQ. Anshul founded and steered PF to become a leading global consultancy specializing in biologics and advanced therapy engineering, with more than 200 professionals. Under Anshul’s leadership, PF pioneered the industrialization of breakthrough therapies, including some of the first commercially approved cell and gene therapies, radioligand treatments, RNA-based therapies and advanced biologics. Anshul’s commitment to advancing next-generation medicine extends from PF to several boards and philanthropic organizations, including IQHQ, Alliance for Regenerative Medicines, Alliance for mRNA Medicines, Institute for Life Changing Medicines and several patient advocacy organizations. Before his tenure in PF, Anshul was a lawyer and founded several digital agencies.

John Khoury is executive vice president at Project Farma. He is a 20+ year veteran and leader in the biotech/pharmaceutical industry. As a member of the Project Pharma and Precision for Medicine leadership teams, John has led the company’s growth in gene and cell therapy. He has led and strategized key partner initiatives including make vs. buy analysis, location and supplier selection, technology transfer execution strategies and facility start-up. In addition to his experience in advanced therapies, John has extensive experience with small and large molecules, including biologics and biosimilars. Over the past four years, he has led the construction of facilities totaling more than $500 million. During this time, he has worked with small and large biotech and pharmaceutical companies, including gene and cell therapy startups and CMOs/CROs.